[co-author: Silvia Lopez Arnao]

The European Medicines Agency (“EMA”) and the EU network of Health Technology Assessment (“HTA”) bodies are jointly reviewing a proposed framework for patient preference studies. The intention of the proposed framework is to provide recommendations to support the development of guidelines concerning the inclusion of patient preferences on the benefit-risk assessment of medicinal products and medical devices. The result of the joint review will be included in the first joint qualification opinion by the EMA and the EU network of HTA bodies. The final qualification opinion is expected to be published by the end of 2021

The European Medicines Agency (“EMA”) and the EU network of Health Technology Assessment (“HTA”) bodies are jointly reviewing a proposed framework for patient preference studies. The intention of the proposed framework is to provide recommendations to support the development of guidelines concerning the inclusion of patient preferences on the benefit-risk assessment of medicinal products and medical devices.

The framework under review

The proposed framework was developed in the context of the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (“PREFER”) research project. The PREFER project is a public-private collaborative research project carried out within the Innovative Medicines Initiative (IMI). This Initiative is the result of a public-private partnership between the European Federation of Pharmaceutical Industries and Associations (“EFPIA”) and the European Commission.

Patient preferences are defined in the proposed framework as the process whereby patients compare and choose one medicinal product or medical device over another by comparing between different options.

The PREFER project provides a structured approach to assessing and using patient preferences during the development, authorisation or conformity assessment, and post-authorisation/CE marking phases of medicinal products and medical devices. A set of principles, guidelines, and tools to be considered when designing, conducting, and analysing the results of a patient preference study are provided. The guidance includes the organisation of the study, research questions, ethics and good practice, and the application of preference data to complement decision-making. The proposed framework also provides guidance to decision-makers when assessing and using preference study results.

The joint qualification opinion

The result of the joint review will be included in the first joint qualification opinion by the EMA and the EU network of HTA bodies.

Patient preferences currently play a limited role in the benefit-risk assessments carried out by regulators and HTA bodies. HTA bodies find patient preference studies useful for investigating the benefits and risks of the administration of a treatment. However, whether this information influences their decision-making depends on the quality of the study, a PREFER study founds. This is where the joint qualification opinion by the EMA and the EU network of HTAs bodies could be helpful. This is because it provides guidance concerning the acceptability of a specific use of a method.

Before the adoption of the joint qualification opinion, the draft framework will be published for public consultation. The final qualification opinion is expected to be published by the end of 2021.

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